Steven U. Walkley

Chronic Cyclodextrin Treatment of Murine Niemann-Pick C Disease Ameliorates Neuronal Cholesterol and Glycosphingolipid Storage and Disease Progression

Cristin Davidson, Nafeeza Ali, Matthew C. Micsenyi et al. · PLoS ONE · 2009 · 449 citations · Full text

Genetic evidence for nonredundant functional cooperativity between NPC1 and NPC2 in lipid transport

David E. Sleat, Jennifer A. Wiseman, Mukarram El-Banna et al. · Proceedings of the National Academy of Sciences · 2004 · 369 citations · Full text

Proteinlipid Interaction, Molecular Biology, Lipid Movement +20

Critical role for glycosphingolipids in Niemann-Pick disease type C

Mark Zervas, Kyra L Somers, Mary Anna Thrall et al. · Current Biology · 2001 · 330 citations · Full text

Autoimmune Disease, Disease Mechanism, G Protein-coupled Receptor +7

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial

Daniel S. Ory, Elizabeth A. Ottinger, Nicole Y. Farhat et al. · The Lancet · 2017 · 316 citations · Full text

Neurodegenerative Diseases, Neurobiology Of Disease, Clinical Therapeutic +9

Neurons in Niemann-Pick Disease Type C Accumulate Gangliosides as Well as Unesterified Cholesterol and Undergo Dendritic and Axonal Alterations

Mark Zervas, Kostantin Dobrenis, Steven U. Walkley · Journal of Neuropathology & Experimental Neurology · 2001 · 266 citations · Full text

Pathology, Axonal Alterations, Cellular Physiology +29